OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.

Condition	Treatment or Intervention
Cystinosis	Drug: cysteamine hydrochloride

Further Study Details: 

Expected Total Enrollment:  30

PROTOCOL OUTLINE: This is a randomized, multicenter study Patients are randomized to receive the current formulation of cysteamine hydrochloride as drops in one eye and the new formulation of cysteamine hydrochloride as drops in the other eye. Patients receive the two formulations of cysteamine hydrochloride in their assigned eyes every hour during waking hours daily for 6 months (safety study) or for 1 year (efficacy study).

Ages Eligible for Study:  1 Year   -   50 Years,  Genders Eligible for Study:  Both

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

• Diagnosis of cystinosis with more than 2 nmole half-cystine/mg protein in leukocytes OR presence of corneal crystals consistent with cystinosis and distributed in corneal stroma observed by slit lamp biomicroscopy
• Clinical history consistent with cystinosis
• Safety study: History of adherence with current eye drop and follow-up schedule on protocol #86-El-0062 Any crystal density score, including zero, on photographs, that has been stable or improved over the past year
• Efficacy study: Crystal density score at least 1.00 on photographs Concurrently on cysteamine for at least the past 6 months

--Prior/Concurrent Therapy--

• No prior cysteamine drops (efficacy study)

--Patient Characteristics--

• Age: 1 to 50 (safety study) 2 to 12 (efficacy study)

- Other: Willingness and ability to tolerate corneal photographs

Study chairs or principal investigators

Edward F. Lemanowicz,  Study Chair,  Sigma-Tau Research, Inc.   

Study ID Numbers:  199/15704; SIGMATAU-FDR001769
Record last reviewed:  April 2001
Last Updated:  October 13, 2004
Record first received:  February 2, 2001
ClinicalTrials.gov Identifier:  NCT00010426
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08